PHILADELPHIA, April 04, 2017 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated gene therapy company dedicated to challenging the inevitability of genetic disease, announced today that Adam Cuker, M.D., assistant professor of medicine at the Perelman School of Medicine of the University of Pennsylvania and a clinical investigator in the Phase 1/2 clinical trial of SPK-9001 for hemophilia B, will present updated interim data as of March 24, 2017 at the Hemostasis and Thrombosis Research Society (HTRS) 2017 Scientific Symposium in Scottsdale, Arizona.
The poster entitled “Initiation of Oral Steroid Therapy Produced Rapid Normalization of Transaminase Levels and Stabilization of FIX Activity in Subjects Who Developed Capsid-Directed T-Cell Responses in the SPK-9001: Adeno-Associated Virus-Mediated Gene Transfer for Hemophilia B Phase 1/2 Trial,” will be presented on Friday, April 7 from 5:15-6:15 p.m. MST.
About Spark Therapeutics
Spark Therapeutics, a fully integrated company, strives to challenge the inevitability of genetic disease by discovering, developing, and delivering gene therapies that address inherited retinal diseases (IRDs), neurodegenerative diseases, as well as diseases that can be addressed by targeting the liver. Our validated platform successfully has delivered proof-of-concept data with investigational gene therapies in the retina and liver. Our most advanced investigational candidate, voretigene neparvovec, in development for the treatment of biallelic RPE65-mediated IRD, has received orphan designations in the U.S. and European Union, and breakthrough therapy designation in the U.S. The pipeline also includes SPK-7001 in a Phase 1/2 trial for choroideremia, and two hemophilia development programs: SPK-9001 (which also has received both breakthrough therapy and orphan product designations by the FDA, and access to the PRIority MEdicines (PRIME) Program by the EMA) in a Phase 1/2 trial for hemophilia B being developed in collaboration with Pfizer, and SPK-8011, in a Phase 1/2 trial for hemophilia A to which Spark Therapeutics retains global commercialization rights. To learn more about us and our growing pipeline, visit www.sparktx.com.